CAR-T for autoimmune disease: what Erlangen proved and what comes next

For the first time in history, a one-time infusion of genetically engineered immune cells — called CAR-T cells — has been shown to put severe, treatment-resistant autoimmune diseases like lupus into complete, drug-free remission. Patients who had failed every existing treatment over many years were, after a single infusion, able to stop all immunosuppressant drugs entirely. The immune system effectively rebooted itself — clearing the disease-causing cells while leaving the patient able to respond normally to vaccines and infections.

The patients didn't just improve. Several achieved drug-free remission — a state that, for many autoimmune conditions, was considered functionally impossible.

Diseases treated to date include Systemic Lupus Erythematosus (SLE), idiopathic inflammatory myositis (IIM), systemic sclerosis (SSc), antiphospholipid syndrome, immune thrombocytopenia (ITP), and autoimmune hemolytic anemia (AIHA).^[1]

What the Trials Showed

The Erlangen approach targeted CD19, a protein found on B cells — the immune cells that, in autoimmune disease, turn against the body's own tissue. By depleting these cells and allowing the immune system to reconstitute itself from scratch, the therapy appeared to reset the underlying pathology rather than merely suppress it, as conventional immunosuppressants do.^[1]

The work itself was the product of more than a decade of groundwork. Schett's lab had been studying B cells and autoantibodies in rheumatic disease for years before CAR-T was shown to be effective in blood cancers like leukemia — a result that suggested the same tool might work on autoimmune disease too.^[3] The first SLE patient was treated cautiously in 2021, given how unknown the safety profile of CAR-T was outside of oncology.^[3] By September 2022, a landmark paper in Nature Medicine reported that all five treated SLE patients had achieved drug-free remission, with the first patient sustaining remission for over 17 months.^[2,3] The results triggered what can only be described as a global biopharma gold rush — by late 2022, more than 30 companies had announced their own autoimmune CAR-T programs.^[3]

The evidence base kept building. By June 2023, all eight SLE patients in the trial had reached DORIS remission with a SLEDAI-2K score of zero — meaning no detectable disease activity at all — while off every immunosuppressant.^[3] A February 2024 case series in the New England Journal of Medicine reported 15 patients across SLE, myositis, and systemic sclerosis all achieving complete remission after a single infusion.^[2,3] Through 2024, the trial expanded into additional autoimmune diseases, with results presented at ACR Convergence.^[3] By 2025, safety and long-term efficacy data existed for more than 30 patients, including the first case of CAR-T used to treat a patient with three simultaneous autoimmune diseases at once.^[3]

Why Bavaria

This breakthrough did not happen at a Boston or Bay Area biotech — it happened at a public university hospital in a mid-sized Bavarian city, and that location is not incidental to the story.

University Hospital Erlangen (Universitätsklinikum Erlangen) sits at Ulmenweg 18 in Erlangen, Bavaria, as part of Friedrich-Alexander-Universität Erlangen-Nürnberg (FAU) — one of Germany's largest research universities, with roughly 40,000 students.^[1] The hospital campus is fully integrated with the university's biomedical research infrastructure, and critically, the CAR-T cells used in the trials are manufactured on-site, in the hospital's own GMP (Good Manufacturing Practice) cell therapy facility.^[1] That single piece of infrastructure was a decisive logistical advantage: it allowed an academic-led trial to proceed without a commercial manufacturing partner, years before any pharmaceutical company had reason to get involved.

Bavaria's regional government also played a direct funding role, supporting biotech and academic medical center infrastructure in the state — one layer in a funding stack that, at every level, was public rather than commercial.^[1]

Lead Researchers

Prof. Dr. Georg Schett served as Principal Investigator. Born in Austria in 1969, he was in his early-to-mid fifties at the time of the breakthrough. He earned his MD from the University of Innsbruck in 1994, completed postdoctoral training in Internal Medicine and Rheumatology at the University of Vienna, spent time at Amgen in the US, and completed his habilitation — Germany's post-doctoral lecturing qualification — in 2003.^[1] He currently serves as Vice President for Research at FAU and as Director of the Department of Medicine 3 for Rheumatology and Immunology at University Hospital Erlangen, a role he has held since 2006.^[1] In 2023 he received the Gottfried Wilhelm Leibniz Prize — Germany's most prestigious science award, given by the DFG — and was named to STAT's STATUS List in 2025.^[1,6] He also discloses advisory roles at Kyverna Therapeutics and Cabaletta Bio, along with relationships with Bristol-Myers Squibb, Janssen, and Novartis.^[1]

He was joined by several co-investigators: Prof. Dr. Andreas Mackensen, Director of the Department of Medicine 5 for Hematology and Oncology, who was responsible for the cellular manufacturing of the CAR-T cells used in treatment; Dr. Fabian Müller, also in Hematology and Oncology, who served as lead author on several key publications including the landmark 2024 NEJM case series; Prof. Dr. Dimitrios Mougiakakos, who led the immunology work and is now at the University of Magdeburg; and Prof. Dr. Gerhard Krönke, who worked in Rheumatology and Immunology and is now at Charité Berlin.^[1]

Funding

The original patient trials were entirely publicly funded academic studies — no pharmaceutical company was involved in the initial breakthrough.^[1] The funding stack behind that work came from several public sources: the German Research Foundation (DFG) was the primary public funder, with Schett serving as PI of the IMMUNOBONE collaborative research center, funded since 2008, which laid the mechanistic groundwork years before the first CAR-T patient was ever treated.^[1] The 2023 Leibniz Prize itself came with €2.5 million for further research, and European Research Council (ERC) grants supported the underlying autoimmunity and B cell biology work.^[1] FAU contributed institutional funding and the GMP manufacturing infrastructure described above, and the Bavarian state government provided regional biotech and academic medical center funding.^[1] Schett's industry advisory roles at Kyverna and Cabaletta represent potential future commercial channels rather than funding for the original academic trials.^[1]

The Regulatory Path

Germany's regulatory framework for advanced therapy medicinal products, or ATMPs — the category CAR-T falls into — involves several distinct bodies. The Paul-Ehrlich-Institut (PEI), Germany's Federal Institute for Vaccines and Biomedicines and the rough equivalent of the FDA's CBER division for biological products, had to approve the Erlangen team's clinical-grade CAR-T cell product before any patient could be treated.^[1,7] The Berlin State Office for Health and Social Affairs (LAGeSo) approved the GMP manufacturing of the cell product at the hospital facility, and FAU's own Ethics Committee conducted the institutional ethics review — the German equivalent of a US IRB.^[1]

The single most important regulatory detail, though, is the Hospital Exemption pathway. Under EU Regulation 1394/2007, academic hospitals in Germany are permitted to manufacture and administer ATMPs under a national hospital exemption, without going through full EMA marketing authorization.^[1,5] This is the specific legal mechanism that allowed Erlangen to proceed with small-scale academic trials outside of a commercial IND-style structure — without it, this breakthrough likely would have required a pharmaceutical partner from day one. Full EMA marketing authorization would still be required for any eventual EU-wide commercial rollout.^[1]

IP, Licensing, and the Commercial Response

The academic work at Erlangen did not itself generate any patented commercial products — it was basic and translational research, conducted without commercial partners. But it triggered an enormous commercial response almost immediately.^[1]

Kyverna Therapeutics, based in Emeryville, California, licensed an exclusive anti-CD19 CAR-T construct from the NIH's National Cancer Institute — specifically from the lab of James N. Kochenderfer, MD — in January 2022, and Schett subsequently joined their scientific advisory board.^[1] Kyverna is backed by Gilead Sciences and Vida Ventures, and its lead candidate is KYV-101.^[1,8] In a separate 2022 deal, Kyverna licensed Intellia Therapeutics' CRISPR/Cas9 platform to develop an allogeneic, "off-the-shelf" version called KYV-201; Intellia received equity and milestone payments while retaining a US co-commercialization option structured around a 50/50 cost-and-revenue split plus low-to-mid-single-digit royalties.^[1]

Cabaletta Bio, based in Philadelphia, is developing its own CD19 CAR-T therapy for autoimmune disease, also with Schett on its advisory board.^[1,9] Major pharmaceutical companies — Novartis, Bristol-Myers Squibb, and Janssen — are all actively pursuing their own autoimmune CAR-T programs directly inspired by the Erlangen results, and BioNTech in Mainz is developing an adjacent mRNA-based immune tolerance approach to autoimmune disease.^[1] By late 2023, more than 30 companies and 70 programs worldwide traced their origin back to this single academic research effort in Bavaria.^[1]

What Comes Next

The question now is scale. CAR-T therapy remains expensive, complex to manufacture, and currently requires highly specialized treatment centers — the same on-site GMP manufacturing advantage that made Erlangen's academic trials possible is exactly what most hospitals lack. As of 2026, trials are actively recruiting and multiple Phase II/III commercial trials are underway globally.^[1] The path from landmark academic trial to standard of care is long, but for the first time in decades, for a category of disease that medicine had largely treated as a matter of lifelong management rather than potential remission, that path now clearly exists.

Sources & Further Reading

All claims in this post are traceable to the sources below.

Key Publications (Primary Sources)

Lead Researcher Profiles & Institutional Sources

Regulatory Sources

Commercial & IP Sources

News and Secondary Sources